PU-H71 receives orphan drug designation for myelofibrosis

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Micrograph showing myelofibrosis
The US Food and Drug Administration (FDA) has granted orphan drug designation to PU-H71 to treat patients with myelofibrosis. The drug specifically targets the epichaperome, a network of high-molecular- weight complexes found in multiple diseases, including cancer and neurologic disorders. These complexes enhance cellular survival, irrespective of tissue of origin or... [Read Article]
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How ruxolitinib reduces thrombosis in MPNs

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Polycythemia vera
Image courtesy of AFIP
Preclinical research helps explain how the JAK1/2 inhibitor ruxolitinib can reduce thrombosis in patients with myeloproliferative neoplasms (MPNs). Experiments revealed a link between JAK2V617F and the formation of neutrophil extracellular traps (NETs), which have been implicated in thrombosis. Researchers found that ruxolitinib reduced NET formation and decreased... [Read Article]
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FDA approves test to diagnose MPNs

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Essential thrombocythemia
The US Food and Drug Administration (FDA) has cleared use of QIAGEN’s ipsogen JAK2 RGQ PCR Kit (ipsogen JAK2 assay) for the diagnosis of all myeloproliferative neoplasms (MPNs). The ipsogen JAK2 assay is a qualitative, in vitro diagnostic test designed to detect the JAK2 V617F/G1849T allele in genomic DNA extracted from... [Read Article]
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Allo-HSCT leads to long-term survival in MF

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Attendees at ASH 2017
Photo courtesy of ASH
© Todd Buchanan 2017
One of the largest single-center studies of fludarabine/melphalan-based allogeneic hematopoietic stem cell transplant (allo-HSCT) for patients with myelofibrosis (MF) shows excellent overall survival (OS) with a low risk of relapse, according to investigators. Allo-HSCT is the only potential curative treatment modality... [Read Article]
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